El Parkinson nuevamente es noticia, lo mejor, BUENAS NOTICIAS. En una oportunidad anterior se dio a conocer la noticia del descubrimiento de nuevos genes responsables de esta condición de salud, si quieres leer más clic aquí; en esta oportunidad la noticia va relacionada ya que al parecer se ha logrado una terapia génica para el tratamiento de lo síntomas del Parkison.
El estudio fue realizado por un grupo de investigadores del Instituto Investigaciones Médicas de New York, que consistió en comparar el tratamiento génico vs. placebo en 22 participantes con Parkinson a "doble ciego".
Según los entendidos en el área, ha sido una investigación muy bien hecha y " meticulosa", que da como resultado una mejora de los síntomas motores. La investigación fue publicada en la Revista " Lancet" y un reportaje completo de la investigación desde sus inicios y fases fue publicado en ElMundo.es
Dentro de todas las investigaciones que se han hecho con respecto a la terapia génica en el Parkinson, ésta es la que tiene mayor importancia en el área debido a su metodología "doble ciego" y lo que ésto significa.
Peter Lewit, en el estudio publicado "The Lancet Oncology", refiere que los estudios demuestran también que:
"La promesa de la terapia génica para los trastornos neurodegenerativos se ha convertido en una realidad".
The Lancet Neurology, Early Online Publication, 17 March 2011
doi:10.1016/S1474-4422(11)70039-4
Cite or Link Using DOI
AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
Dr Andrew Feigin MD l 
Summary
Background
Gene transfer of glutamic acid decarboxylase (GAD) and other methods that modulate production of GABA in the subthalamic nucleus improve basal ganglia function in parkinsonism in animal models. We aimed to assess the effect of bilateral delivery of AAV2-GAD in the subthalamic nucleus compared with sham surgery in patients with advanced Parkinson's disease.
Methods
Patients aged 30—75 years who had progressive levodopa-responsive Parkinson's disease and an overnight off-medication unified Parkinson's disease rating scale (UPDRS) motor score of 25 or more were enrolled into this double-blind, phase 2, randomised controlled trial, which took place at seven centres in the USA between Nov 17, 2008, and May 11, 2010. Infusion failure or catheter tip location beyond a predefined target zone led to exclusion of patients before unmasking for the efficacy analysis. The primary outcome measure was the 6-month change from baseline in double-blind assessment of off-medication UPDRS motor scores. This trial is registered with ClinicalTrials.gov, NCT00643890.
Findings
Of 66 patients assessed for eligibility, 23 were randomly assigned to sham surgery and 22 to AAV2-GAD infusions; of those, 21 and 16, respectively, were analysed. At the 6-month endpoint, UPDRS score for the AAV2-GAD group decreased by 8·1 points (SD 1·7, 23·1%; p<0·0001) and by 4·7 points in the sham group (1·5, 12·7%; p=0·003). The AAV2-GAD group showed a significantly greater improvement from baseline in UPDRS scores compared with the sham group over the 6-month course of the study (RMANOVA, p=0·04). One serious adverse event occurred within 6 months of surgery; this case of bowel obstruction occurred in the AAV2-GAD group, was not attributed to treatment or the surgical procedure, and fully resolved. Other adverse events were mild or moderate, likely related to surgery and resolved; the most common were headache (seven patients in the AAV2-GAD groupvs two in the sham group) and nausea (six vs two).
Interpretation
The efficacy and safety of bilateral infusion of AAV2-GAD in the subthalamic nucleus supports its further development for Parkinson's disease and shows the promise for gene therapy for neurological disorders.
Funding
Neurologix.
The Lancet Neurology, Early Online Publication, 17 March 2011
doi:10.1016/S1474-4422(11)70039-4Cite or Link Using DOI
Cite or Link Using DOIAAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
Dr Andrew Feigin MD l
Summary
Background
Gene transfer of glutamic acid decarboxylase (GAD) and other methods that modulate production of GABA in the subthalamic nucleus improve basal ganglia function in parkinsonism in animal models. We aimed to assess the effect of bilateral delivery of AAV2-GAD in the subthalamic nucleus compared with sham surgery in patients with advanced Parkinson's disease.
Methods
Patients aged 30—75 years who had progressive levodopa-responsive Parkinson's disease and an overnight off-medication unified Parkinson's disease rating scale (UPDRS) motor score of 25 or more were enrolled into this double-blind, phase 2, randomised controlled trial, which took place at seven centres in the USA between Nov 17, 2008, and May 11, 2010. Infusion failure or catheter tip location beyond a predefined target zone led to exclusion of patients before unmasking for the efficacy analysis. The primary outcome measure was the 6-month change from baseline in double-blind assessment of off-medication UPDRS motor scores. This trial is registered with ClinicalTrials.gov, NCT00643890.
Findings
Of 66 patients assessed for eligibility, 23 were randomly assigned to sham surgery and 22 to AAV2-GAD infusions; of those, 21 and 16, respectively, were analysed. At the 6-month endpoint, UPDRS score for the AAV2-GAD group decreased by 8·1 points (SD 1·7, 23·1%; p<0·0001) and by 4·7 points in the sham group (1·5, 12·7%; p=0·003). The AAV2-GAD group showed a significantly greater improvement from baseline in UPDRS scores compared with the sham group over the 6-month course of the study (RMANOVA, p=0·04). One serious adverse event occurred within 6 months of surgery; this case of bowel obstruction occurred in the AAV2-GAD group, was not attributed to treatment or the surgical procedure, and fully resolved. Other adverse events were mild or moderate, likely related to surgery and resolved; the most common were headache (seven patients in the AAV2-GAD groupvs two in the sham group) and nausea (six vs two).
Interpretation
The efficacy and safety of bilateral infusion of AAV2-GAD in the subthalamic nucleus supports its further development for Parkinson's disease and shows the promise for gene therapy for neurological disorders.
Funding
Neurologix.
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